Brainscape's Knowledge GenomeTM


Top Flashcards (Certified)
All Flashcards

Therapeutics III > Sickle Cell Disease - Block 4 > Flashcards

Sickle Cell Disease - Block 4 Flashcards

1
Q

What is the difference betweent SCT and SCD?

A

SCT: Heterozygous inheritance of one HbA gene and one sickle gene (HbAS)
SCD: Homozygous HbS (HbSS)

How well did you know this?
1
Not at all
2
3
4
5
Perfectly
2
Q

What is the cause of SCD?

A

Gene mutation of b-subunits on hemeglobin (HbS)

How well did you know this?
1
Not at all
2
3
4
5
Perfectly
3
Q

What are the classifications of anemia?

A

Male: Hb <13
Women: Hb <12

How well did you know this?
1
Not at all
2
3
4
5
Perfectly
4
Q

Describe the patho of SCD?

A
  1. Oxygenated HbS is similar to normal RBC
  2. Deoxygenated HbS: reduce solubility and increased binding
  3. Polymeization: distortion of RBC and loss of deformability
  4. Sickled RBCs: increase viscisty and sludging
  5. Reoxygenated: polymer lost and return to normal shape
  6. Cycle: damage to cells, decreased membrane flexibility, and phospholipid rearrangement
  7. Irreversible sickled cell: decreased flow and increased vascular endothelial adherence
  8. Intravascular destruction: 10-20 days
How well did you know this?
1
Not at all
2
3
4
5
Perfectly
5
Q

How are newborns screened for SCD?

A
  1. Isoelectric finding
  2. High performance liquid chromatography
  3. Hb electrophoresis
  • With + creening have 2nd test before 2 months of age to confirm
How well did you know this?
1
Not at all
2
3
4
5
Perfectly
6
Q

When is the onset of SCD in newborns?

A

4-6 month after birth due to prior presence of HbF (2a, 2y)
* 3-4 months RBC life

How well did you know this?
1
Not at all
2
3
4
5
Perfectly
7
Q

Lab findings of SCD in newborns?

A
  1. Low Hb (6-9)
  2. Elevated reticulocytes of 10-25%, platelets and WBC
  3. Normal MCV
  4. Sickle cell
How well did you know this?
1
Not at all
2
3
4
5
Perfectly
8
Q

Burden of SCD in children?

A
  1. Dacylitis before 1 YO
  2. Hb<7
  3. Leokocytosis w/o infection
  4. Rectilocytosis -> stroke and death
  5. Acute chest syndrome first 3 Y
  6. SCD + Asthma -> increaed frequency of ACS
How well did you know this?
1
Not at all
2
3
4
5
Perfectly
9
Q

Burden of disease in adults?

A

Decreased survival:
1. chest pain
2. ElevatedWBC
3. CV event
4. Renal failure
5. Proteinuria
6. Pulmonary HTN

How well did you know this?
1
Not at all
2
3
4
5
Perfectly
10
Q

What are the goals of SC tx?

A

Reduce hospitalization, mortality, and complications

How well did you know this?
1
Not at all
2
3
4
5
Perfectly
11
Q

Types of health maintenance of SCD?

A
  1. Immunization
  2. Invasive pneumococcal infection prevention
  3. Renal
  4. Pulmonary HTN
  5. Ophthalmological evaluation
  6. Stroke prevention
How well did you know this?
1
Not at all
2
3
4
5
Perfectly
12
Q

How should SCD patients recieve immunization?

A

Fall under anatomical or functional asplenia due to impair spleen function

How well did you know this?
1
Not at all
2
3
4
5
Perfectly
13
Q

How is invasive pnemococcal infection prevented?

A

Penicillin prophylaxis till 5 YO who pevent with HbSS or HbSb0-thal until they are completely vaccinated against Pneuococcal
* Pen V K+ 125 mg PO BID until 3YO, 250 BID till 5YO

  • Erythromycin 20mg/kg/day
How well did you know this?
1
Not at all
2
3
4
5
Perfectly
14
Q

How do we screen for pulmonary hypertension?

A
  1. Noninvasive tests (Doppler or serum NT-pro-BNP) -> assess mortality
  2. Pulmonary function test
  3. Polycomnography
  4. Oxygenation assessment
  5. Thromboembolic dx
How well did you know this?
1
Not at all
2
3
4
5
Perfectly
15
Q

Indications for Doppler echo?

A

To screen for PAH and associated cardiac problems by age 8 with frequent cardiorespiratory sx

Recommended Q1-3Y

How well did you know this?
1
Not at all
2
3
4
5
Perfectly
16
Q

Tx for SCD-induced PAH?

A

Cardiac catheterization prior to tx:

How well did you know this?
1
Not at all
2
3
4
5
Perfectly
17
Q

Screenign and tx for renal complications from SCD?

A

Screen: proteinuria by age 10 and annually if negative
Tx: Initiate ACEI for adults with microalbuminuria (30-300) or proteinuria (>300) without apparent cause

How well did you know this?
1
Not at all
2
3
4
5
Perfectly
18
Q

Screenign for opthalmological complications from SCD?

A

Eye exam at 10 YO and rescreen Q1-2Y

How well did you know this?
1
Not at all
2
3
4
5
Perfectly
19
Q

Screenign and tx for stroke from SCD children?

A

Screening: transcranial doppler annually beginning age 2 until 16
Tx: Chronic transfusion therapy for stroke prevention in children with elevated TCD (>200 cm/s)

How well did you know this?
1
Not at all
2
3
4
5
Perfectly
20
Q

WHat is the purpose of HbF induction?

A

Increased HbF -> decreased RBC sickling and adhesion -> Less severe SCD and complications

How well did you know this?
1
Not at all
2
3
4
5
Perfectly
21
Q

Examples of HbF inducation tx?

A
  1. Hydroxyurea (Droxia, Siklos)
  2. L-Glutamine (endari)
  3. Voxelotor (Oxbyrta)
How well did you know this?
1
Not at all
2
3
4
5
Perfectly
22
Q

What is the MOA of hydroxyurea?

A
  1. Stimulates HbF production, reticulocyted, and intracellular HbF
  2. Cytotoxic -> stimulates erythropoisis in bone marrow
  3. Increase NO (vasodilation)
  4. Decrease RBC adhesion to endothelium
How well did you know this?
1
Not at all
2
3
4
5
Perfectly
23
Q

Who qualifies for hydroyurea tx?

A

Adults with SCD:
1. Three or more sicke cell associated mod-severe pain crises per year
2. SCD interferes with ADLs
3. Hx of severe or recurrent ACS
4. Severe symptomatic chronic anemia that affect ADL
5. Infants (9 months) with SCD
6. CKD on eyrthropien

How well did you know this?
1
Not at all
2
3
4
5
Perfectly
24
Q

Hydroxyurea brands or interchangable?

A

No

How well did you know this?
1
Not at all
2
3
4
5
Perfectly
25
Hydroxyurea | DOsing, BBW
**Dosing:** renal dosing (<60) - 5-10mg/kg * Admin at the same time everyday * ANtineoplastic requires proper handeling **BBW** bone marrow suppression (leukopenia, neutropenia, thrombocytopenia, anemia, decreased reticulocyte count) * Reproductive tox * Carcinogenic
26
MOA of L-Glutamine
Restores redox balance in oxidative stressed cell by NAD+ synthesis * reduced pain episodes and hospitalizations
27
ADR of L-glutamine?
N, C, noncardiac chest pain, fatigue, musculoskeletal pain
28
Voxelotor | MOA, Indication, DDI, ADR
**MOA:** HbS polymerization inhibitor -> stabilized the oxygenated Hb state **Indication:** ≥12YO **DDI:** 1,500 mg taken once daily unless weight is under 40 kg; dose adjustment to 1,000 mg once daily in severe hepatic impairment * CYP3A4 substrate **ADR:** HA, D
29
What is the function of chronic transfusion?
**Purpose:** prevent primary and secondary stroke prevention and improve organ damage **Method:** simple transfusion, manual and automated exchange **Benefit:** increase normal HbA, decrease viscosity, limit volume **Cons:** Iron overload, alloimmunization, volume overload, infection, reaction, hyperviscosity
30
How often are transfusions given?
3-4 weeks but is adjusted to maintain desired HbS levels
31
What is the transfusion goal?
1. HbS concentration <30% 2. absence of recurrent stroke for 2 years, Hbs <50%
32
How do we minimize the risk of transfusion?
1. Hep A and B vaccine 2. Blood screening (virus) 3. Cross matched blood products (phenotyping) 4. Avoid excess dietary iron
33
What is DHTR?
Occurs 7-10 days after transfusion Sx: hemolysis -> ab pain, anemia, reticulocytopenia
34
Tx of DHTR?
1. Steroids 2. IV immunoglobin 3. rErythropoietin (reticulocytopenia) 4. Rituximab
35
How do you diagnose transfusional iron overload?
1. LFTs and serum ferritin (annual or semi) 2. Liver biposy (inflammation or fibrosis) 3. MRI
36
Tx for iron overload?
**SC or IV:** Deferoxamine **Oral:** Deferosirox, Deferiprone
37
ADR of deferasirox?
**BBW:** GI hemmorrhage, hepatic injury, AKI **ADR:** skin rash, GI
38
ADR of deferiprone?
**BBW:** agranulocytosis, neutropenia **ADR:** urine discoloration, nausea
39
Biomarkers for cirrhosis?
1. Increased INR, PTT 2. Increased biirubin 3. Decreased albumin and platelets
40
Biomarkers for acute hepatic injury?
Increase AST, ALT, ALP
41
What is the only curative tx for SCD?
Allogenic hematopoietic stem cell transplantation: used prior to organ damage and alloimmunization and screen for HLA identical sibling first year of life
42
Who qualifies for Allogeneic Hematopoietic Stem Cell Transplantation?
1. Children and young adults with sibling matched donors 2. For unrelated matched allogeneic HSCT, 16 yo or younger with severe SCD and complications
43
Complications for Allogeneic Hematopoietic Stem Cell Transplantation?
1. GVHD 2. Sz 3. Marrow rejection 4. Sepsis 5. Death
44
What re the presentations of fever?
Fever > 38.5°C (101.3°F) -> determine the risk of infection Fever ≥  39.5°C (103.1°F) and ill appearance -> hospitalization
45
WHat is aplastic crisis?
Decrease in reticulocyte count and rapid development of severe anemia
46
What is the criteria for hospitalization for fever and infection?
1. <1YO 2. Hx of previous bacteremia or sepsis 3. 103.1°F 4. WBB >30 5. Platelet <100
47
Medications for tx fever and infection?
**ABX:** * Ceftriaxone/cefotaxime * Clindamycin (for those with cephalosporin allergy) * Vancomycin (if acutely ill or Staph suspected) * Macrolide (Mycoplasma pneumoniae [ACS]) **Antipyretics:** APAP, IBU **FLuids:**
48
What are vasocclusive processes?
CV occulusion -> stroke and ischemia
49
What are signs of severe anemia?
1. Lower intelligence 2. Visual motor impairments 3. Neurophysical dysfunctions
50
Common sign for hemorhagic stroke?
Convulsions
51
How do we evaluate neuroligc complications for SCD?
**Acute:** exam (Q2Y), CT, MRI **Screeing:** MRI, TCD, EEG
52
Wht is the tx for neurological problems?
**Acute:** hospitalization and monitoring **Children:** exchange transfusion tomaintain Hgb at 10g/dL and HbS <30% * Anticonvulsants -> sz * Tx for increased ICP (mannitol, hypertonic solution) **Adults:** Determine if due to SCD or atherosclerotic disease
53
What is acute schest syndrome?
New pulmonary infiltrate with one or more of the following: cough, dyspnea, tachypnea, chest pain, fever, wheezing, and new-onset hypoxia
54
How is most at risk for ACS?
Young age
55
Describe the severity of ACS?
Hypoxia, pulmonary changes (pleural effusions), bilateral infiltrates/multi-lobe involvement
56
How do we evaluate ACS?
1. PE 2. CBC 3. Chest x-ray Severe: CT, perfusion scintigraphy, transthoracic echocardiography, bronchoscopy
57
What is the tx for ACS?
1. Incentive spirometry (Q2H) 2. Pain managemetn 3. Fluid 4. Broad spec abx (macorlide or quinolone) 5. O2 6. Bronchodilator 7. Transfusions 8. Steroids 9. NO
58
What is the difference between stuttering and ischemic priapism?
**Stuttering:** repeated intermittant attacks up to several hours before remission **Ischemic:** persistent painful erection >4hr (emergency)
59
What is the non pharm for priapism?
1. Exercise, hydration, heat (hot water bottles, hot packs, sitz baths) 2. Aspiration 3. Surgically placed shunts (severe refractory)
60
What is the pharm for priapism?
1. Analgesic 2. Vasoconstrictors to force blood from the corpus cavernosum into venous return (Diluted PE or E) 3. Vasodilator to relax smooth muscle of the vasculature (terbutaline and hydralazine)
61
How can we prevent priapism?
1. PE 2. Leuprolide 3. Hydroxyurea 4. Antiandrogen (bicalutamide, finasteride)
62
What is the location of most SC pain?
Back, chest, extremities
63
What do we manage acute SCD pain?
**Mild-mod:** nonopioid +/- weak opioid **Mod-severe:** weak opioid or low dose opioid +/- nonopioid **Severe:** strong opioid + nonopioid
64
What are the add on for SCD pain?
1. Hydration, O2, heating pads, relaxation, distraction 2. Laxatives 3. Antihistamine 4. Antiemetics
65
What are the types of analgesics?
66
Tx for vaso-occlusive crises?
Crizanlizumab (Adakveo)
67
What is Splenic sequestration?
Sudden massive enlargement of the spleen resulting from the sequestration of sickled RBCs in the splenic parenchyma
68
How do you evaluate for splenic sequestration?
1. Close monitoring of vital signs 1. Spleen size 1. Oxygen saturation 1. CBC 1. Reticulocyte count 1. Cultures
69
WHat is the tx for splenic sequestration?
1. Fluid resuscitation 1. Blood transfusions 1. Broad-spectrum antibiotics (if indicated) 1. Splenectomy
70
What is the screening tool for VTE?
D-dimer
71
What is the diagnosis for PAH?
Pulmonary artery pressure (PAP) > 25 mmHg using doppler
72
How do we tx PAH in SCD?
1. Hydroxyurea 2. Chronic transfusion 3. PDE5i
73
How often do you get eye exams?
Anually
74
How often do you do med evals?
* ≤1YO: every 2-4 months * ≥ 2YO: every 6-12 months with modifications depending on severity
75
Tests used for med eval?
1. TCD starting 2YOU is HbSS is presnet 2. PFTs for patients with recurrent ACS 2. Ophthalmologic exam for retinopathy * 10-12 YOU if HbSC * 14 for HbSS
76
How often do you monitor CBC?
< 2YO: every 3-6 months ≥ 2 YO: every 6-12 months
77
How often do you monitor HbF?
Annually until 2 YO
78
How are we evaluating in ABX tx?
1. Prevention of pneumococcal infection 2. Clinical improvement when ill
79
Tx eval for hydroxyurea?
1. Decrease in the number, severity, and duration of sickle cell pain episodes 2. HbF concentrations or MCV values
80
Tx eval for analgesics?
* Patient reported relief * Non-verbal pain cues ameliorated

Therapeutics III (40 decks)

Brainscape helps you reach your goals faster, through stronger study habits.
© 2025 Bold Learning Solutions. Terms and Conditions